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ECM‐Related Myopathies and Muscular Dystrophies: Pros and Cons of Protein Therapies

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ABSTRACT

Extracellular matrix (ECM) myopathies and muscular dystrophies are a group of genetic diseases caused by mutations in genes encoding proteins that provide critical links between muscle cells and the extracellular matrix. These include structural proteins of the ECM, muscle cell receptors, enzymes, and intracellular proteins. Loss of adhesion within the myomatrix results in progressive muscle weakness. For many ECM muscular dystrophies, symptoms can occur any time after birth and often result in reduced life expectancy. There are no cures for the ECM‐related muscular dystrophies and treatment options are limited to palliative care. Several therapeutic approaches have been explored to treat muscular dystrophies including gene therapy, gene editing, exon skipping, embryonic, and adult stem cell therapy, targeting genetic modifiers, modulating inflammatory responses, or preventing muscle degeneration. Recently, protein therapies that replace components of the defective myomatrix or enhance muscle and/or extracellular matrix integrity and function have been explored. Preclinical studies for many of these biologics have been promising in animal models of these muscle diseases. This review aims to summarize the ECM muscular dystrophies for which protein therapies are being developed and discuss the exciting potential and possible limitations of this approach for treating this family of devastating genetic muscle diseases. © 2017 American Physiological Society. Compr Physiol 7:1519‐1536, 2017.

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Figure 1. Figure 1. Muscular dystrophies and myopathies associated with the myomatrix. Model of the extracellular matrix, dystrophin associated glycoprotein complex and α7β1 integrin complex in skeletal muscle. DMD and BMD are caused by mutations that result in a complete (DMD) or a partial (BMD) loss of dystrophin. LGMD type 2D, 2E, 2C, and 2F results from deficiencies in the components of the sarcoglycan complex. LGMD 2B and Miyoshi myopathy are caused by mutations in the dysferlin gene. LAMA2‐CMD is a congenital muscular dystrophy characterized by the loss of laminin 211 and 221 isoforms. Collagen VI‐related CMD is caused by mutations in COL6A1, COL6A2, and COL6A3 genes. ITGA7‐related CMD is caused by mutations in the α7 integrin gene.
Figure 2. Figure 2. Biglycan, laminin‐111 and mini‐agrin protein therapies for the treatment of the muscular dystrophies. (A) Biglycan therapy stabilizes the membrane through extracellular matrix binding of dystroglycans and sarcoglycans, which leads to utrophin upregulation in DMD. (B) Laminin‐111 therapy is a laminin‐211 replacement therapy where the adult laminin isoform () is replaced by the embryonic isoform () which binds to integrin and dystroglycans leading to membrane stabilization and regeneration in LAMA2‐CMD. (C) Mini‐agrin is a miniature version of agrin, which links laminin 411 to the dystroglycans ameliorating membrane stability in LAMA2‐CMD.
Figure 3. Figure 3. Growth factor therapies can effectively ameliorate pathology in LAMA2‐CMD and DMD. (A) Insulin‐like growth factor‐1 (IGF‐1) binds to insulin‐like growth factor receptor 1 (IGF‐1R), which promotes muscle regeneration by regulating proliferation, differentiation, and fusion of myoblasts to existing muscle fibers. (B) Losartan is an angiotensin II receptor type 1 (AT1) antagonist associated with lowering levels of transforming growth factor‐β (TGF‐β). High levels of TGF‐β are associated with increased fibrosis and inhibition of myogenesis.
Figure 4. Figure 4. TAT‐utrophin, MG53 and Galectin‐1 protein therapies. (A) TAT‐Utrophin is a replacement therapy where utrophin (full length or mini‐utrophin) is used as the protein therapy to stabilize the protein complex UGC. (B) MG53 is an endogenous endoplasmic protein that in response to oxidative environment forms a complex with caveolin 3, dysferlin, annexin V, and PTRF causing the oligomerization of vesicles and consequently membrane repair. (C) Galectin‐1 interacts with laminin and α7β1 integrin promoting myoblast fusion during muscle repair.
Figure 5. Figure 5. Wnt7a protein therapy induces satellite stem cell proliferation in muscle. Recombinant Wnt7a protein promotes satellite cell expansion leading to larger muscle fibers and a switch from fast to slow muscle fiber types resulting in increased force of dystrophic muscle. Wnt7a protein therapy also induces polarity of satellite stem cells allowing migration to already formed muscle fibers and better engraftment of transplanted cells.


Figure 1. Muscular dystrophies and myopathies associated with the myomatrix. Model of the extracellular matrix, dystrophin associated glycoprotein complex and α7β1 integrin complex in skeletal muscle. DMD and BMD are caused by mutations that result in a complete (DMD) or a partial (BMD) loss of dystrophin. LGMD type 2D, 2E, 2C, and 2F results from deficiencies in the components of the sarcoglycan complex. LGMD 2B and Miyoshi myopathy are caused by mutations in the dysferlin gene. LAMA2‐CMD is a congenital muscular dystrophy characterized by the loss of laminin 211 and 221 isoforms. Collagen VI‐related CMD is caused by mutations in COL6A1, COL6A2, and COL6A3 genes. ITGA7‐related CMD is caused by mutations in the α7 integrin gene.


Figure 2. Biglycan, laminin‐111 and mini‐agrin protein therapies for the treatment of the muscular dystrophies. (A) Biglycan therapy stabilizes the membrane through extracellular matrix binding of dystroglycans and sarcoglycans, which leads to utrophin upregulation in DMD. (B) Laminin‐111 therapy is a laminin‐211 replacement therapy where the adult laminin isoform () is replaced by the embryonic isoform () which binds to integrin and dystroglycans leading to membrane stabilization and regeneration in LAMA2‐CMD. (C) Mini‐agrin is a miniature version of agrin, which links laminin 411 to the dystroglycans ameliorating membrane stability in LAMA2‐CMD.


Figure 3. Growth factor therapies can effectively ameliorate pathology in LAMA2‐CMD and DMD. (A) Insulin‐like growth factor‐1 (IGF‐1) binds to insulin‐like growth factor receptor 1 (IGF‐1R), which promotes muscle regeneration by regulating proliferation, differentiation, and fusion of myoblasts to existing muscle fibers. (B) Losartan is an angiotensin II receptor type 1 (AT1) antagonist associated with lowering levels of transforming growth factor‐β (TGF‐β). High levels of TGF‐β are associated with increased fibrosis and inhibition of myogenesis.


Figure 4. TAT‐utrophin, MG53 and Galectin‐1 protein therapies. (A) TAT‐Utrophin is a replacement therapy where utrophin (full length or mini‐utrophin) is used as the protein therapy to stabilize the protein complex UGC. (B) MG53 is an endogenous endoplasmic protein that in response to oxidative environment forms a complex with caveolin 3, dysferlin, annexin V, and PTRF causing the oligomerization of vesicles and consequently membrane repair. (C) Galectin‐1 interacts with laminin and α7β1 integrin promoting myoblast fusion during muscle repair.


Figure 5. Wnt7a protein therapy induces satellite stem cell proliferation in muscle. Recombinant Wnt7a protein promotes satellite cell expansion leading to larger muscle fibers and a switch from fast to slow muscle fiber types resulting in increased force of dystrophic muscle. Wnt7a protein therapy also induces polarity of satellite stem cells allowing migration to already formed muscle fibers and better engraftment of transplanted cells.
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Pam M. Van Ry, Tatiana M. Fontelonga, Pamela Barraza‐Flores, Apurva Sarathy, Andreia M. Nunes, Dean J. Burkin. ECM‐Related Myopathies and Muscular Dystrophies: Pros and Cons of Protein Therapies. Compr Physiol 2017, 7: 1519-1536. doi: 10.1002/cphy.c150033